ArticleNucleic acids research2018
Designer epigenome modifiers enable robust and sustained gene silencing in clinically relevant human cells.
Article in Nucleic acids research, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.
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Who cites it
62 citing papers in PubMed, 101 citations in OpenAlex.
- From epigenetic mark detection to rational design of epigenetic editing strategies.Molecular therapy. Advances · 2026Review
- Beyond Permanent Genome Editing: Molecular Delivery Strategies for RNA Editing and Epigenome-Editing Therapeutics.International journal of molecular sciences · 2026Review
- Advances and clinical potential of epigenome editing.Cellular and molecular life sciences : CMLS · 2026Review
- Epigenetic editing: from concept to clinic.Nature reviews. Drug discovery · 2026Review
- Advances and challenges of precision epigenetic therapy in treating genomic imprinting diseases.Translational pediatrics · 2026Review
- Toward designer magic bullets: Epigenetic editing to empower CAR T cells.Molecular therapy. Nucleic acids · 2025Article
- Engineering Inducible Cell Fate Transitions by Harnessing Epigenetic Silencing.bioRxiv : the preprint server for biology · 2025Article
- Sustained and specific multiplexed immune checkpoint modulation in CAR T cells induced by targeted epigenome editing.Molecular therapy. Nucleic acids · 2025Article
- Dissecting the epigenetic regulation of the fetal hemoglobin genes to unravel a novel therapeutic approach for β-hemoglobinopathies.Nucleic acids research · 2025Article
- Epigenetic editing and epi-drugs: a combination strategy to simultaneously target KDM4 as a novel anticancer approach.Clinical epigenetics · 2025Article
- Gene regulation technologies for gene and cell therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Delta Opioid Peptide [d-Ala2, d-Leu5]-Enkephalin Improves Physical and Cognitive Function and Increases Lifespan in Aged Female Mice.Molecular neurobiology · 2025Article
- The Promise of Epigenetic Editing for Treating Brain Disorders.Sub-cellular biochemistry · 2025Review
- Generating mRNA Encoding Anti-HBV Designer Epigenome Modifiers.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Epigenetic mechanisms in cardiovascular complications of diabetes: towards future therapies.Molecular medicine (Cambridge, Mass.) · 2024Review
- The therapeutic implications of all-in-one AAV-delivered epigenome-editing platform in neurodegenerative disorders.Nature communications · 2024Article
- Brainwide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor.Science (New York, N.Y.) · 2024Article
- Keep Fingers on the CpG Islands.Epigenomes · 2024Article
- Effects of DNA methylation and its application in inflammatory bowel disease (Review).International journal of molecular medicine · 2024Review
- Hit-and-run epigenome editing durably lowers cholesterol in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
2 more citing papers are in PubMed but not listed here.
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Targeted modulation of gene expression represents a valuable approach to understand the mechanisms governing gene regulation. In a therapeutic context, it can be exploited to selectively modify the aberrant expression of a disease-causing gene or to provide the target cells with a new function. Here, we have established a novel platform for achieving precision epigenome editing using designer epigenome modifiers (DEMs). DEMs combine in a single molecule a DNA binding domain based on highly specific transcription activator-like effectors (TALEs) and several effector domains capable of inducing DNA methylation and locally altering the chromatin structure to silence target gene expression. We designed DEMs to target two human genes, CCR5 and CXCR4, with the aim of epigenetically silencing their expression in primary human T lymphocytes. We observed robust and sustained target gene silencing associated with reduced chromatin accessibility, increased promoter methylation at the target sites and undetectable changes in global gene expression. Our results demonstrate that DEMs can be successfully used to silence target gene expression in primary human cells with remarkably high specificity, paving the way for the establishment of a potential new class of therapeutics.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.